FDA Approves First Treatment for Patients with Cerebral Folate Transport Deficiency
WHY IT MATTERS
For the first time, patients with confirmed FOLR1 variants now have an FDA-approved treatment option specifically designed for cerebral folate deficiency, moving this condition from having no approved therapy to having a targeted treatment available.
The FDA has approved a new use for a medicine called Wellcovorin (leucovorin calcium) to treat a rare condition called cerebral folate deficiency. This condition happens when people have a genetic change in a gene called FOLR1 that prevents their brain from getting enough folate, a B vitamin it needs to work properly. This approval means adults and children with this specific genetic change can now use this medicine as a treatment.
The U.S. Food and Drug Administration today approved expanded use of Wellcovorin (leucovorin calcium) tablets for the treatment of cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1).
YOU CAN ACT ON THIS
If you or your child has been diagnosed with cerebral folate deficiency with a FOLR1 variant, contact your neurologist or metabolic specialist immediately to discuss whether Wellcovorin is appropriate and how to access it.